Posted by Michael Wonder on 25 Mar 2026
FDA approves drug to treat neurologic manifestations of Hunter syndrome
25 March 2026 - The US FDA approved Avlayah (tividenofusp alfa-eknm) to treat certain individuals with Hunter syndrome (mucopolysaccharidosis type II or MPS II).
To support approval, the sponsor submitted results from a Phase 1/2 multi-cohort, single-arm, open-label trial that enrolled 47 paediatric patients with Hunter syndrome aged 3 months to 13 years.
